Thursday, Jul 7, 2016

FDA Approves Genentech’s Xolair^® (omalizumab) for Allergic Asthma in Children

• Approval makes Xolair the only biologic for children ages six and up with uncontrolled allergic asthma
• Asthma is one of the most common long-term diseases in children¹, affecting about 6.3 million people younger than 18 in the U.S.²
• More than 200,000 allergic asthma patients have been treated with Xolair since its approval in 2003 for people 12 years and older³

South San Francisco, CA -- July 7, 2016 --

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) approved Xolair^® (omalizumab) to treat moderate to severe persistent asthma in children six to 11 years of age who have had a positive skin test or in vitro reactivity to an airborne allergen and have symptoms that are inadequately controlled with inhaled corticosteroids⁴. Xolair is already approved to treat people 12 years and older with allergic asthma.

"Despite our best efforts to control symptoms with inhaled corticosteroids and other medicines, allergic asthma remains a serious problem for many children," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "With this approval, we’re pleased to see a proven treatment option is now available for appropriate patients six and older."

Asthma is one of the most common long-term diseases in children¹. It affects about 6.3 million people under 18 or one in 12 children in the U.S ². An estimated 24 million people in the U.S. have asthma. Of this patient population, approximately 60 percent have allergic asthma⁵ . The American Academy of Pediatrics estimates that between 70 and 80 percent of school-aged children with asthma also have allergies, which are among the most common triggers for asthma⁶.

"Uncontrolled allergic asthma can significantly affect the lives of children," said Cary Sennett, M.D., PhD, president and chief executive officer of the Asthma and Allergy Foundation of America (AAFA). "This approval helps to address an important unmet need for children older than six and their parents or caregivers."

Xolair was first approved in 2003 to treat adults and children 12 years of age and older with moderate to severe persistent allergic asthma not controlled by inhaled steroids. Since its U.S. approval, more than 200,000 patients older than 12 with allergic asthma have been treated with the medicine³ . Xolair is not indicated for the treatment of other allergic conditions, acute bronchospasm (serious and sudden breathing problems) or status asthmaticus (acute, severe, prolonged asthma attack that can be life-threatening).

The latest approval is supported by multi-center, randomized, double-blind, placebo-controlled Phase III studies that assessed the efficacy and safety of Xolair in children from six to 11 years old with moderate to severe persistent uncontrolled allergic asthma. The primary study was a 52-week trial, with the primary endpoint measured at 24 weeks⁴. Supportive safety and efficacy data come from a 28-week study⁴. Additional safety data come from a five-year non-randomized observational post-marketing study to evaluate the long-term safety of Xolair in patients 12 years and older ⁷.

Efficacy and Safety Findings

The 52-week study evaluated the safety and efficacy of Xolair as an add-on therapy in children from six to 11 years old with moderate to severe allergic asthma who were inadequately controlled despite the use of inhaled corticosteroids with or without the use of other controller asthma medications. During the first 24 weeks of treatment, steroid doses remained constant from baseline. This was followed by a 28-week period during which inhaled corticosteroid adjustment was allowed. The primary efficacy variable in this study was the rate of asthma exacerbations during the 24-week, fixed steroid treatment phase. An asthma exacerbation was defined as a worsening of asthma symptoms as judged clinically by the investigator, requiring doubling of the baseline inhaled corticosteroid dose for at least three days and/or treatment with rescue systemic corticosteroids for at least three days⁴.

At 24 weeks, the Xolair treatment group had a statistically significantly lower rate of asthma exacerbations compared to the placebo treatment group (0.45 vs. 0.64, respectively), representing a 31 percent relative rate reduction (rate ratio 0.69, 95 percent CI (0.53, 0.90) p=0.007)⁴. During the entire 52-week treatment period, the difference in asthma exacerbation rates between the Xolair and placebo treatment groups (0.78 vs. 1.36, respectively) represented a 43 percent relative rate reduction (rate ratio 0.57, 95 percent CI (0.45, 0.72) p<0.001)⁴.

In clinical studies with pediatric patients six to 11 years old, the most common side effects (≥3 percent in Xolair-treated patients and more frequent than placebo) were common cold symptoms (nasopharyngitis), headache, fever (pyrexia), upper abdominal pain, sore throat (pharyngitis streptococcal), ear discomfort (otitis media), intestinal infection causing abdominal pain, nausea and vomiting (viral gastroenteritis), insect bites (arthropod bites) and nose bleeding (epistaxis)⁴.

In the U.S., Genentech, Inc. and Novartis Pharmaceuticals Corporation work together to develop and co-promote Xolair.

Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets innovative medicines aimed at improving patients’ lives.

About Allergic Asthma

Asthma is a chronic condition with inflammation and narrowing of the airways, as well as tightening of the muscles around the airways.

The symptoms of asthma and allergic asthma are the same—it's the triggers that are different. What makes allergic asthma a unique type of asthma is the cause of its symptoms: exposure to year-round allergens in the air, such as pet dander and dust mites. If you have allergic asthma, allergens like these can bring on your allergic asthma symptoms and attacks.

• When an allergen enters your body, your immune system identifies it as something harmful;
• Your immune system responds by releasing a substance called immunoglobulin E, or IgE;
• IgE plays a key role in the development of allergic asthma symptoms. It binds to allergens, which causes the release of chemicals that can lead to swelling (inflammation) in and around the lungs. This can trigger an allergic asthma attack.

People with allergic asthma may have higher levels of IgE because of the way their immune system reacts to allergens. For some, blocking IgE, which Xolair is designed to do, has been shown to be a helpful part of their allergic asthma treatment plan. If you think you may have allergic asthma, ask your doctor about how much IgE is in your body.

About Xolair

Xolair^® for subcutaneous use is an injectable prescription medicine used to treat:

• moderate to severe persistent asthma in patients six years of age or older whose asthma symptoms are not controlled by asthma medicines called inhaled corticosteroids. A skin or blood test is performed to see if a patient has allergies to year-round allergens.
• chronic idiopathic urticaria (CIU; chronic hives without a known cause) in patients 12 years of age and older who continue to have hives that are not controlled by H1 antihistamine treatment.

Xolair is not used to treat other allergic conditions, other forms of urticaria, acute bronchospasm or status asthmaticus.

IMPORTANT SAFETY INFORMATION

The most important information patients should know about Xolair is that a severe allergic reaction called anaphylaxis can happen when a patient receives Xolair. The reaction can occur after the first dose, or after many doses. It may also occur right after a Xolair injection or days later. Anaphylaxis is a life-threatening condition and can lead to death. Patients must go to the nearest emergency room right away if they have any of these symptoms of an allergic reaction:

• wheezing, shortness of breath, cough, chest tightness, or trouble breathing
• low blood pressure, dizziness, fainting, rapid or weak heartbeat, anxiety, or feeling of “impending doom”
• flushing, itching, hives, or feeling warm
• swelling of the throat or tongue, throat tightness, hoarse voice, or trouble swallowing

The patient’s healthcare provider will monitor the patient closely for symptoms of an allergic reaction while they are receiving Xolair and for a period of time after the patient’s injection. The patient’s healthcare provider should talk to the patient about getting medical treatment if they have symptoms of an allergic reaction after leaving the healthcare provider’s office or treatment center.

Patients must not receive Xolair if they are allergic to omalizumab or any of the ingredients in Xolair.

Before receiving Xolair, patients must tell their healthcare provider about all of their medical conditions, including if they:

• have any other allergies (such as food allergy or seasonal allergies)
• have sudden breathing problems (bronchospasm)
• have ever had a severe allergic reaction called anaphylaxis
• have or have had a parasitic infection
• have or have had cancer
• are pregnant or plan to become pregnant. It is not known if Xolair may harm a patient’s unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if Xolair passes into breast milk. Talk with your healthcare provider about the best way to feed your baby while you receive Xolair.

Patients must tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, or herbal supplements.

Receiving Xolair

• Xolair should be given by a healthcare provider, in a healthcare setting.
• Xolair is given in one or more injections under the skin (subcutaneous), one time every two or four weeks.
• In asthma patients, a blood test for a substance called IgE must be performed prior to starting Xolair to determine the appropriate dose and dosing frequency.
• In patients with chronic hives, a blood test is not necessary to determine the dose or dosing frequency.
• Patients must not decrease or stop taking any of their other asthma or hive medicine unless their healthcare providers tell them to.
• Patients may not see improvement in their symptoms right away after Xolair treatment.

Possible side effects of Xolair

Xolair may cause serious side effects, including:

• See, “What is the most important information I should know about Xolair” in the Xolair Medication Guide at http://www.xolair.com regarding the risk of anaphylaxis.
• Cancer. Cases of cancer were observed in some people who received Xolair.
• Inflammation of your blood vessels. Rarely, this can happen in people with asthma who receive Xolair. This usually, but not always, happens in people who also take a steroid medicine by mouth that is being stopped or the dose is being lowered. It is not known whether this is caused by Xolair. Tell your healthcare provider right away if you have rash; chest pain; shortness of breath; or a feeling of pins and needles or numbness of your arms or legs.
• Fever, muscle aches, and rash. Some people who take Xolair get these symptoms one to five days after receiving a Xolair injection. If a patient has any of these symptoms, they must tell their healthcare provider.
• Parasitic infection. Some people who are at a high risk for parasite (worm) infections, get a parasite infection after receiving Xolair. The patient’s healthcare provider can test the patient’s stool to check if they have a parasite infection.
• Heart and circulation problems. Some people who receive Xolair have had chest pain, heart attack, blood clots in the lungs or legs, or temporary symptoms of weakness on one side of the body, slurred speech, or altered vision. It is not known whether this is caused by Xolair.

The most common side effects of Xolair:

• In adults and children 12 years of age and older with asthma: pain especially in the arms and legs, dizziness, feeling tired, skin rash, bone fractures and pain or discomfort of the ears.
• In children 6 to less than 12 years of age with asthma: common cold symptoms, headache, fever, sore throat, pain or discomfort of your ear, abdominal pain, nausea, vomiting and nose bleeds.
• In people with chronic idiopathic urticaria: nausea, headaches, swelling of the inside of the nose, throat or sinuses, cough, joint pain and upper respiratory tract infection.

These are not all the possible side effects of Xolair. Patients should call their doctor for medical advice about side effects.

Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch.com. Report side effects to Genentech at (888) 835-2555 or Novartis Pharmaceuticals Corporation at (888) 669-6682.

Please see full Prescribing Information, including Medication Guide at http://www.xolair.com for additional Important Safety Information.

 

About Xolair Access Solutions

Patients experiencing financial hardship may be eligible to receive coverage and reimbursement support for Xolair. Patient assistance and informational resources are available through Xolair Access Solutions (http://www.xolair.com/allergic-asthma/xolair-access-solutions.html).

About Genentech

Founded 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

References

¹ U.S. Centers for Disease Control and Prevention. What Is Asthma? http://www.cdc.gov/asthma/faqs.htm. Accessed June 30, 2016.

² U.S. Centers for Disease Control and Prevention. National Current Asthma Prevalence. http://www.cdc.gov/asthma/most_recent_data.htm. Accessed June 21, 2016.

³ Data on file. Genentech, Inc, South San Francisco, CA.

⁴ Xolair^® Full Prescribing Information. Genentech. July 7, 2016.

⁵ Arbes S, Gergen P, Vaughn B, et al. Asthma cases attributable to atopy: Results from the Third National Health and Nutrition Examination Survey. J Allergy Clin Immunol 2007;120:1139-45.

⁶ American Academy of Pediatrics. Healthy Children.org. Guide to Your Child’s Allergies and Asthma. https://www.healthychildren.org/English/health-issues/conditions/allergies-asthma/Pages/Allergies.aspx. Accessed June 30, 2016.

⁷ Long A, Rahmaoui A, Rothman K, et al. Incidence of malignancy in patients with moderate-to-severe asthma treated with or without omalizumab. J Allergy Clin Immunol. 2014;134:560-7.

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